RT Journal Article
T1 A combination of ascorbic acid and α-tocopherol to test the effectiveness and safety in the fragile X syndrome: study protocol for a phase II, randomized, placebo-controlled trial.
A1 de Diego-Otero, Yolanda
A1 Calvo-Medina, Rocio
A1 Quintero-Navarro, Carolina
A1 Sánchez-Salido, Lourdes
A1 García-Guirado, Francisco
A1 del Arco-Herrera, Ignacio
A1 Fernández-Carvajal, Isabel
A1 Ferrando-Lucas, Teresa
A1 Caballero-Andaluz, Rafaela
A1 Pérez-Costillas, Lucia
K1 Antioxidants
K1 Experimental treatment
K1 Fragile X syndrome
K1 Oxidative stress
K1 Trial
K1 Síndrome del cromosoma X frágil
K1 Ácido ascórbico
K1 Alfa-tocoferol
K1 España
K1 Resultado del tratamiento
K1 Masculino
AB BACKGROUNDFragile X syndrome (FXS) is an inherited neurodevelopmental condition characterised by behavioural, learning disabilities, physical and neurological symptoms. In addition, an important degree of comorbidity with autism is also present. Considered a rare disorder affecting both genders, it first becomes apparent during childhood with displays of language delay and behavioural symptoms.Main aim: To show whether the combination of 10 mg/kg/day of ascorbic acid (vitamin C) and 10 mg/kg/day of α-tocopherol (vitamin E) reduces FXS symptoms among male patients ages 6 to 18 years compared to placebo treatment, as measured on the standardized rating scales at baseline, and after 12 and 24 weeks of treatment.Secondary aims: To assess the safety of the treatment. To describe behavioural and cognitive changes revealed by the Developmental Behaviour Checklist Short Form (DBC-P24) and the Wechsler Intelligence Scale for Children-Revised. To describe metabolic changes revealed by blood analysis. To measure treatment impact at home and in an academic environment.METHODS/DESIGNA phase II randomized, double-blind pilot clinical trial.SCOPEmale children and adolescents diagnosed with FXS, in accordance with a standardized molecular biology test, who met all the inclusion criteria and none of the exclusion criteria.INSTRUMENTATIONclinical data, blood analysis, Wechsler Intelligence Scale for Children-Revised, Conners parent and teacher rating scale scores and the DBC-P24 results will be obtained at the baseline (t0). Follow up examinations will take place at 12 weeks (t1) and 24 weeks (t2) of treatment.DISCUSSIONA limited number of clinical trials have been carried out on children with FXS, but more are necessary as current treatment possibilities are insufficient and often provoke side effects. In the present study, we sought to overcome possible methodological problems by conducting a phase II pilot study in order to calculate the relevant statistical parameters and determine the safety of the proposed treatment. The results will provide evidence to improve hyperactivity control and reduce behavioural and learning problems using ascorbic acid (vitamin C) and α-tocopherol (vitamin E). The study protocol was approved by the Regional Government Committee for Clinical Trials in Andalusia and the Spanish agency for drugs and health products.TRIAL REGISTRATIONClinicalTrials.gov Identifier: NCT01329770 (29 March 2011).
PB BioMed Central
YR 2014
FD 2014-09-03
LK http://hdl.handle.net/10668/2095
UL http://hdl.handle.net/10668/2095
LA en
NO de Diego-Otero Y, Calvo-Medina R, Quintero-Navarro C, Sánchez-Salido L, García-Guirado F, del Arco-Herrera l, et al. A combination of ascorbic acid and α-tocopherol to test the effectiveness and safety in the fragile X syndrome: study protocol for a phase II, randomized, placebo-controlled trial. Trials. 2014; 15:345
NO Clinical Trial, Phase II; Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't;
DS RISalud
RD Apr 14, 2025